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Huntington’s Disease Therapeutic Targets Found

By staff

3/19/2014

US Pharm. 2014;39(3):49-50.

A study led by researchers at Boston University School of Medicine (BUSM) offers insight into the impact that genes may have on Huntington’s disease (HD). The study, published online in PLOS Genetics, identified specific small segments of RNA (called micro RNA or miRNA) encoded in DNA in the human genome that are highly expressed in HD. The researchers showed that these miRNAs are present in higher quantities in patients with HD and may act as a mitigating factor in the neurologic decline associated with the disease, making them a possible therapeutic target.

“If this miRNA were also found outside of brain tissue, for example in the blood, it could be used as an inexpensive, non-invasive assessment of the severity of the disease and perhaps for evaluating the effectiveness of treatments in clinical trials for HD. If the amount of miRNA were quantified in an HD patient, the amount could provide insight into the likely age of disease onset or life expectancy of the patient which current genetic testing in HD does not provide,” explained lead author Richard Myers, PhD, professor of neurology at BUSM.

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